Phase I Study of APX005M in Pediatric Central Nervous System Tumors · Source last updated 2026-08-13
Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.
Measure
MEAN
Unit
μg/mL
Interval / dispersion
Standard Deviation
Time frame
Up to 12 weeks from start of study drug
What was measured
Serial blood samples for APX005M pharmacokinetic studies were collected during courses 1 and 2 at pre-dose, at the end of infusion, and at 4, 24 ± 1 (day 2), and 168 ± 4 hours (day 8) from the start of infusion in that course. During courses 3 and 4, samples were obtained pre-dose and end of induction. Serum concentrations of sotigalimab were measured using a validated electrochemiluminescent (ECL) immunoassay (Method ICD 853 v1.00). Mean serum concentrations with standard deviations were calculated using Phoenix® WinNonlin® v.8.4. Concentrations below the limit of quantitation (0.010 μg/mL) were replaced with 0 to calculate means and SDs.
Analysis population: Ineligible patients were excluded from all analyses.
Groups in this outcome
Stratum 1, Dose Level 1
Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.1 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
Stratum 1, Dose Level 2
Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
Stratum 1, Dose Level 3
Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
Stratum 2, Dose Level 2
Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
Stratum 2, Dose Level 3
Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
3
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
13
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
5
Reported measurements
Course 1, pre-dose
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
3
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
13
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
4
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
0
0
Not reported
Not reported
Not reported
Course 1, 1 hour
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
3
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
12
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
3
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0.120
0.115
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
3.95
0.526
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
9.15
2.98
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
3.49
0.984
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
9.87
1.95
Not reported
Not reported
Not reported
Course 1, 4 hours
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
1
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
12
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
3
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0.0357
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
2.29
0.349
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
6.64
1.93
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
2.12
1.06
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
7.88
0.280
Not reported
Not reported
Not reported
Course 1, 24 hours
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
3
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
8
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
3
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
0.851
1.44
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
0.0136
0.0137
Not reported
Not reported
Not reported
Course 1, 168 hours
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
3
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
13
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
3
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
0
0
Not reported
Not reported
Not reported
Course 2, pre-dose
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
3
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
11
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
2
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
1.26
2.19
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
0
0
Not reported
Not reported
Not reported
Course 2, 1 hour
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
3
Stratum 1, Dose Level 2
2
Stratum 1, Dose Level 3
11
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
2
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0.148
0.135
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
4.10
0.247
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
8.47
2.27
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
3.23
1.48
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
9.63
2.22
Not reported
Not reported
Not reported
Course 2, 4 hours
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
2
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
11
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
2
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0.032
0.0253
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
2.66
0.140
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
6.13
1.87
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
1.55
0.959
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
7.41
3.15
Not reported
Not reported
Not reported
Course 2, 24 hours
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
3
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
7
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
2
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
0.593
0.872
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
0.0317
0.0448
Not reported
Not reported
Not reported
Course 2, 168 hours
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
3
Stratum 1, Dose Level 2
3
Stratum 1, Dose Level 3
10
Stratum 2, Dose Level 2
6
Stratum 2, Dose Level 3
2
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
0
0
Not reported
Not reported
Not reported
Course 3, pre-dose
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
1
Stratum 1, Dose Level 2
2
Stratum 1, Dose Level 3
4
Stratum 2, Dose Level 2
4
Stratum 2, Dose Level 3
2
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
0
0
Not reported
Not reported
Not reported
Course 3, 1 hour
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
1
Stratum 1, Dose Level 2
2
Stratum 1, Dose Level 3
4
Stratum 2, Dose Level 2
4
Stratum 2, Dose Level 3
2
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0.198
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
3.54
0.721
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
7.33
1.98
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
1.89
1
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
9.53
1.80
Not reported
Not reported
Not reported
Course 4, pre-dose
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
1
Stratum 1, Dose Level 2
1
Stratum 1, Dose Level 3
4
Stratum 2, Dose Level 2
4
Stratum 2, Dose Level 3
1
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
0
0
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
0
0
Not reported
Not reported
Not reported
Course 4, 1 hour
Analysis denominator · Participants
Group
Source count
Stratum 1, Dose Level 1
1
Stratum 1, Dose Level 2
1
Stratum 1, Dose Level 3
4
Stratum 2, Dose Level 2
3
Stratum 2, Dose Level 3
1
Values in μg/mL · Interval/dispersion: Standard Deviation
Group
Value
Spread
Lower
Upper
Comment
Stratum 1, Dose Level 1
0.0114
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 2
3.98
0
Not reported
Not reported
Not reported
Stratum 1, Dose Level 3
8.82
3.11
Not reported
Not reported
Not reported
Stratum 2, Dose Level 2
1.26
1.17
Not reported
Not reported
Not reported
Stratum 2, Dose Level 3
1.84
0
Not reported
Not reported
Not reported
Complete source fields
classes
categories
measurements
group Id
OG000
spread
0
value
0
group Id
OG001
spread
0
value
0
group Id
OG002
spread
0
value
0
group Id
OG003
spread
0
value
0
group Id
OG004
spread
0
value
0
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
13
group Id
OG003
value
6
group Id
OG004
value
4
units
Participants
title
Course 1, pre-dose
categories
measurements
group Id
OG000
spread
0.115
value
0.120
group Id
OG001
spread
0.526
value
3.95
group Id
OG002
spread
2.98
value
9.15
group Id
OG003
spread
0.984
value
3.49
group Id
OG004
spread
1.95
value
9.87
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
12
group Id
OG003
value
6
group Id
OG004
value
3
units
Participants
title
Course 1, 1 hour
categories
measurements
group Id
OG000
spread
0
value
0.0357
group Id
OG001
spread
0.349
value
2.29
group Id
OG002
spread
1.93
value
6.64
group Id
OG003
spread
1.06
value
2.12
group Id
OG004
spread
0.280
value
7.88
denoms
counts
group Id
OG000
value
1
group Id
OG001
value
3
group Id
OG002
value
12
group Id
OG003
value
6
group Id
OG004
value
3
units
Participants
title
Course 1, 4 hours
categories
measurements
group Id
OG000
spread
0
value
0
group Id
OG001
spread
0
value
0
group Id
OG002
spread
1.44
value
0.851
group Id
OG003
spread
0
value
0
group Id
OG004
spread
0.0137
value
0.0136
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
8
group Id
OG003
value
6
group Id
OG004
value
3
units
Participants
title
Course 1, 24 hours
categories
measurements
group Id
OG000
spread
0
value
0
group Id
OG001
spread
0
value
0
group Id
OG002
spread
0
value
0
group Id
OG003
spread
0
value
0
group Id
OG004
spread
0
value
0
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
13
group Id
OG003
value
6
group Id
OG004
value
3
units
Participants
title
Course 1, 168 hours
categories
measurements
group Id
OG000
spread
0
value
0
group Id
OG001
spread
2.19
value
1.26
group Id
OG002
spread
0
value
0
group Id
OG003
spread
0
value
0
group Id
OG004
spread
0
value
0
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
11
group Id
OG003
value
6
group Id
OG004
value
2
units
Participants
title
Course 2, pre-dose
categories
measurements
group Id
OG000
spread
0.135
value
0.148
group Id
OG001
spread
0.247
value
4.10
group Id
OG002
spread
2.27
value
8.47
group Id
OG003
spread
1.48
value
3.23
group Id
OG004
spread
2.22
value
9.63
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
2
group Id
OG002
value
11
group Id
OG003
value
6
group Id
OG004
value
2
units
Participants
title
Course 2, 1 hour
categories
measurements
group Id
OG000
spread
0.0253
value
0.032
group Id
OG001
spread
0.140
value
2.66
group Id
OG002
spread
1.87
value
6.13
group Id
OG003
spread
0.959
value
1.55
group Id
OG004
spread
3.15
value
7.41
denoms
counts
group Id
OG000
value
2
group Id
OG001
value
3
group Id
OG002
value
11
group Id
OG003
value
6
group Id
OG004
value
2
units
Participants
title
Course 2, 4 hours
categories
measurements
group Id
OG000
spread
0
value
0
group Id
OG001
spread
0
value
0
group Id
OG002
spread
0.872
value
0.593
group Id
OG003
spread
0
value
0
group Id
OG004
spread
0.0448
value
0.0317
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
7
group Id
OG003
value
6
group Id
OG004
value
2
units
Participants
title
Course 2, 24 hours
categories
measurements
group Id
OG000
spread
0
value
0
group Id
OG001
spread
0
value
0
group Id
OG002
spread
0
value
0
group Id
OG003
spread
0
value
0
group Id
OG004
spread
0
value
0
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
10
group Id
OG003
value
6
group Id
OG004
value
2
units
Participants
title
Course 2, 168 hours
categories
measurements
group Id
OG000
spread
0
value
0
group Id
OG001
spread
0
value
0
group Id
OG002
spread
0
value
0
group Id
OG003
spread
0
value
0
group Id
OG004
spread
0
value
0
denoms
counts
group Id
OG000
value
1
group Id
OG001
value
2
group Id
OG002
value
4
group Id
OG003
value
4
group Id
OG004
value
2
units
Participants
title
Course 3, pre-dose
categories
measurements
group Id
OG000
spread
0
value
0.198
group Id
OG001
spread
0.721
value
3.54
group Id
OG002
spread
1.98
value
7.33
group Id
OG003
spread
1
value
1.89
group Id
OG004
spread
1.80
value
9.53
denoms
counts
group Id
OG000
value
1
group Id
OG001
value
2
group Id
OG002
value
4
group Id
OG003
value
4
group Id
OG004
value
2
units
Participants
title
Course 3, 1 hour
denoms
counts
group Id
OG000
value
3
group Id
OG001
value
3
group Id
OG002
value
13
group Id
OG003
value
6
group Id
OG004
value
5
units
Participants
description
Serial blood samples for APX005M pharmacokinetic studies were collected during courses 1 and 2 at pre-dose, at the end of infusion, and at 4, 24 ± 1 (day 2), and 168 ± 4 hours (day 8) from the start of infusion in that course. During courses 3 and 4, samples were obtained pre-dose and end of induction. Serum concentrations of sotigalimab were measured using a validated electrochemiluminescent (ECL) immunoassay (Method ICD 853 v1.00). Mean serum concentrations with standard deviations were calculated using Phoenix® WinNonlin® v.8.4. Concentrations below the limit of quantitation (0.010 μg/mL) were replaced with 0 to calculate means and SDs.
dispersion Type
Standard Deviation
groups
description
Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.1 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG000
title
Stratum 1, Dose Level 1
description
Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG001
title
Stratum 1, Dose Level 2
description
Patients with recurrent or refractory primary malignant CNS tumors received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG002
title
Stratum 1, Dose Level 3
description
Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.3 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG003
title
Stratum 2, Dose Level 2
description
Patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG) received APX005M on day 1 of each 21-day course at 0.6 mg/kg for up to 36 courses or until disease progression, unacceptable toxicity, or death.
id
OG004
title
Stratum 2, Dose Level 3
param Type
MEAN
population Description
Ineligible patients were excluded from all analyses.
Triangle